This clinical research study is being done to determine how safe and effective an experimental drug called sodium oxybate is in reducing excessive daytime sleepiness and cataplexy in subjects with narcolepsy. The experimental drug is a controlled-release formulation of sodium oxybate and only has to be taken once a day. It is a powder supplied in a sachet (or sachets) that is mixed with water and taken at bedtime.

Who can participate?

People 16 years or older with a diagnosis of narcolepsy who experience:

o Excessive daytime sleepiness AND cataplexy

The study:

If you are eligible for this study, the duration of your participation will be 17 weeks. This includes nine visits to the study clinic.

If you do not live close to a study site but are both willing to participate and match the study eligibility criteria, reasonable travel costs can be covered. This will be assessed on a case-by-case basis.

Your suitability for study participation will be assessed at the first study visit and throughout a three-week screening period. If eligible to continue in the study, you will have seven additional study visits during the treatment and follow-up period.

You will be required to take the study drug at bedtime each night at home and follow all instructions given to you by the study staff.

The hospital research team will plan your first and all subsequent visit dates together with you in order to best fit your personal schedule.

If you qualify for the study, you will receive comprehensive care via regular health checks from a dedicated narcolepsy team, trial medication, and compensation for travel- and study-related costs.

Flamel Ireland Ltd. has established a new extended-release formulation of sodium oxybate utilising its specialist Micropump® technology. This Micropump® technology has been approved in both the US and EU for both carvedilol and aspirin. The application of Micropump® to sodium oxybate allows for this slow release of the drug into the body. Therefore, patients will only need to take a single dose at bedtime.

Flamel Ireland Ltd. is currently running a Phase III clinical trial to evaluate the drug’s safety and effectiveness in patients with narcolepsy.

Flamel Ireland Ltd. (Flamel), a wholly owned subsidiary of Flamel Technologies SA, is a specialty pharmaceutical company with a long history of expertise in drug delivery with a range of drug delivery platforms. Flamel’s proprietary technology is designed to extend and/or delay the absorption of drugs, which allows for amore controlled regulation of the drug’s PK profile. Micropump® is one of its delivery platforms, based on a microparticulate system that allows the development and marketing of modified and/or controlled dosage formulations of drugs (Micropump®-carvedilol and Micropump®-aspirin formulations have been approved in the US and in the EU, respectively).

Flamel Ireland Ltd. are currently running a phase III clinical trial to evaluate once-daily sodium oxybate’s safety and effectiveness in patients with narcolepsy.

The Patient Voice:

Anchoring the needs and perspectives of patients in key trial development activities is important to Flamel. The value added by patient engagement is that it allows for clinical trial conduct that is more relevant and meaningful to the needs of study patients and ultimately the clinical population. To help achieve the goal of patient engagement, Flamel has partnered with the Narcolepsy Network (USA) and has established a specialist Patient Advisory Group (PAG) for this clinical trial. The inaugural meeting for the PAG was held in April 2016, followed by a second meeting at the annual Narcolepsy Network Conference in Orlando.

This partnership will help to facilitate a structured approach to patient engagement for the planned Phase III clinical trial.

Founding Principals of the Flamel PAG

* Anchoring the needs and perspectives of patients in key trial development activities.

* Recognition of the importance and critical value of a patient-centric approach in clinical trial development activities.

* Creating greater awareness and understanding across the broader narcolepsy patient community concerning the importance of, the need for and the benefits of clinical research.

Why is this study being done?

This clinical research study is being done to understand how safe and effective the study drug is in reducing excessive daytime sleepiness and cataplexy in subjects with narcolepsy compared to a placebo.

What stage is this research study at?

This is a Phase III clinical research study. Phase III trials look at how well a new treatment works and examine any side effects it may have. Sodium oxybate is currently used as a treatment for narcolepsy and has undergone clinical trials to show that it is safe and effective. This clinical research study is being done to measure the safety and effectiveness of the new extended-release formulation of sodium oxybate.

How long is the study?

The study will take place over 17 weeks. You will have to visit the study clinic nine times.

What if I do not live close to a participating study site? Will my travel costs be covered?

If you do not live close to a study site but are both willing to participate and match the study eligibility criteria, reasonable travel costs can be covered. This will be assessed on a case-by-case basis.

If I take part in this study, what will I need to do?

You will need to:

Complete a Daily Sleep Diary.

Take your study medication each night.

Answer a weekly questionnaire.

Continue to take your stimulants (if applicable).

Keep track of your health and well-being and share this information with your study doctor at your next visit.

Keep note of any medication taken (e.g. for a cold, or supplements such as Vitamin C).

Keep note of any visits to other healthcare facilities.

There are some medications that you will not be permitted to take if you participate in this clinical research study. Your study doctor will discuss these medications with you. If you have cataplexy, you will have to stop all medications that are prescribed to you for treatment of your cataplexy symptoms. Your study doctor will monitor your health closely during the study while you are not taking these medications.

PLEASE NOTE: IF YOU PARTICIPATE IN THIS RESEARCH STUDY YOU CAN REMAIN ON YOUR STIMULANTS FOR THE DURATION OF THE STUDY.

Do I have to take part in the study?

Taking part in this study is your choice. There will not be any penalty or loss of benefits to you if you decide not to take part or if you leave the study early. You may leave the study at any time.

Who will have access to my information if I participate?

Information collected from you will be kept confidential. Your identity will be protected. Only a study number will be used to identify your study information. No information that could identify you will leave your doctor’s site.

For the purposes of review and audit, your study information can be accessed by:

Your study doctor and site staff;

Flamel, the study sponsor, and representatives working on Flamel’s behalf for the purposes of this study;

IRB/EC and regulatory bodies.

Anyone who has access to your information is bound to protect your identity.

2. Have you ever been diagnosed with one of the following: Sleep Apnea, Epilepsy, past history of seizures, Hepatitis B or Hepatitis C?

3. Have you ever taken sodium oxybate? If yes, select when you last took this drug.

Within last month

Within last 6 months

More than 6 months ago

Have Never Taken Sodium Oxybate

4. How long have you been experiencing excessive daytime sleepiness?

Less than 1 month

Less than 3 months

More than 3 months

More than 1 year

Cataplexy is a sudden loss of muscle strength or control that may be triggered by heightened emotions. A cataplexy attack may range in scale from a slackening of the facial muscles or dropping of the jaw to complete loss of muscle control resulting in buckling of the knees or full collapse.

5. Do you experience Cataplexy?

5a. If yes, do you tend to use any non-medical methods, to try and control your cataplexy? Methods such as, avoiding situations which may trigger a cataplexy attack, for example watching a funny movie.

5b. If yes, how often do experience cataplexy attacks, on average per week?

Less than 2 times per week

2-4 times per week

4-6 times per week

7-10 times per week

More than 11 times per week

5c. If yes, do you currently take any prescription medication to manage your Cataplexy?

5d. If yes, would you consider stopping this medication for the duration of the study?

6. This study requires you to attend 9 pre-scheduled study visits over a 17-week period. Site staff will discuss with you how best to plan these visits in advance, to best suit your schedule. Can you please outline below how else the study site staff may assist you in adhering to the schedule and attending all required visits.

7. Only for females: Are you pregnant or breastfeeding?

8. Do you consume alcohol?

8a. If yes, are you willing to refrain from drinking alcohol for the duration of the study if you are selected to participate?

9. Do you drive for a living?

10. Do you work night or rotating shifts?

11. Are you willing to record your sleep and symptoms daily on an electronic device, which will be provided for the purpose of this study?

How did you hear about us?

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